PL3361869T3 - Cząsteczki kwasu nukleinowego zawierające odstępniki i sposoby ich zastosowania - Google Patents
Cząsteczki kwasu nukleinowego zawierające odstępniki i sposoby ich zastosowaniaInfo
- Publication number
- PL3361869T3 PL3361869T3 PL16856281.7T PL16856281T PL3361869T3 PL 3361869 T3 PL3361869 T3 PL 3361869T3 PL 16856281 T PL16856281 T PL 16856281T PL 3361869 T3 PL3361869 T3 PL 3361869T3
- Authority
- PL
- Poland
- Prior art keywords
- methods
- nucleic acid
- acid molecules
- molecules containing
- containing spacers
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2800/00—Nucleic acids vectors
- C12N2800/70—Vectors containing special elements for cloning, e.g. topoisomerase, adaptor sites
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2800/00—Nucleic acids vectors
- C12N2800/90—Vectors containing a transposable element
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562241483P | 2015-10-14 | 2015-10-14 | |
| PCT/US2016/057055 WO2017066579A1 (en) | 2015-10-14 | 2016-10-14 | Nucleic acid molecules containing spacers and methods of use thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL3361869T3 true PL3361869T3 (pl) | 2026-04-27 |
Family
ID=58518407
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL16856281.7T PL3361869T3 (pl) | 2015-10-14 | 2016-10-14 | Cząsteczki kwasu nukleinowego zawierające odstępniki i sposoby ich zastosowania |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US11230720B2 (pl) |
| EP (1) | EP3361869B8 (pl) |
| AU (3) | AU2016338565B2 (pl) |
| CA (1) | CA3001594A1 (pl) |
| PL (1) | PL3361869T3 (pl) |
| PT (1) | PT3361869T (pl) |
| WO (1) | WO2017066579A1 (pl) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3001594A1 (en) * | 2015-10-14 | 2017-04-20 | Audentes Therapeutics, Inc. | Nucleic acid molecules containing spacers and methods of use thereof |
| DK3768846T5 (da) | 2018-03-21 | 2024-08-19 | Aldevron L L C | Virale og ikke-virale nanoplasmidvektorer med forbedret produktion |
| JP2023535632A (ja) | 2020-07-27 | 2023-08-18 | アンジャリウム バイオサイエンシズ エージー | Ｄｎａ分子の組成物、その作製方法、及びその使用方法 |
| US20230265455A1 (en) * | 2020-07-29 | 2023-08-24 | University Of Florida Research Foundation, Incorporated | Improved aav-mediated x-linked retinoschisis therapies |
| EP4504953A4 (en) * | 2022-04-05 | 2026-04-15 | Astellas Gene Therapies Inc | DOSAGE OF ADENE-ASSOCIATED VIRUS AND ITS USES |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6080569A (en) | 1993-06-24 | 2000-06-27 | Merck & Co., Inc. | Adenovirus vectors generated from helper viruses and helper-dependent vectors |
| AU722375B2 (en) | 1996-09-06 | 2000-08-03 | Trustees Of The University Of Pennsylvania, The | Methods using cre-lox for production of recombinant adeno-associated viruses |
| US6346415B1 (en) | 1997-10-21 | 2002-02-12 | Targeted Genetics Corporation | Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors |
| AU3642601A (en) | 1999-11-05 | 2001-05-30 | Avigen, Inc. | Ecdysone-inducible adeno-associated virus expression vectors |
| US20020182595A1 (en) | 2001-04-27 | 2002-12-05 | Weitzman Matthew D. | Method of identifying cellular regulators of adeno-associated virus (AAV) |
| US8067156B2 (en) | 2001-05-31 | 2011-11-29 | The Rockefeller University | Method for generating replication defective viral vectors that are helper free |
| US7419817B2 (en) | 2002-05-17 | 2008-09-02 | The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. | Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography |
| US20040137626A1 (en) | 2002-09-26 | 2004-07-15 | Greenville Hospital System | AAV ITR-mediated modulation |
| WO2006036502A2 (en) | 2004-09-22 | 2006-04-06 | St. Jude Children's Research Hospital | Improved expression of factor ix in gene therapy vectors |
| EP2524037B1 (en) | 2010-01-12 | 2018-05-16 | The University Of North Carolina At Chapel Hill | Restrictive inverted terminal repeats for viral vectors |
| US20110269735A1 (en) * | 2010-04-19 | 2011-11-03 | Celera Corporation | Genetic polymorphisms associated with statin response and cardiovascular diseases, methods of detection and uses thereof |
| US9249425B2 (en) * | 2011-05-16 | 2016-02-02 | The Trustees Of The University Of Pennslyvania | Proviral plasmids and production of recombinant adeno-associated virus |
| TWI698240B (zh) | 2012-05-15 | 2020-07-11 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| AU2013336601B2 (en) | 2012-10-26 | 2018-01-25 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| HK1220488A1 (zh) | 2013-03-15 | 2017-05-05 | The Children's Hospital Of Philadelphia | 含有填充者/填充物多核苷酸序列的载体及其使用方法 |
| FR3004463A1 (fr) * | 2013-04-11 | 2014-10-17 | Genethon | Systeme d'expression pour une therapie genique selective |
| JP6700180B2 (ja) * | 2013-10-28 | 2020-05-27 | アイカーン スクール オブ メディスン アット マウント サイナイＩｃａｈｎ Ｓｃｈｏｏｌ ｏｆ Ｍｅｄｉｃｉｎｅ ａｔ Ｍｔ． Ｓｉｎａｉ | 神経興奮性および運動行動を調節するための組成物および方法 |
| CN107427557B (zh) | 2014-08-13 | 2022-01-04 | 费城儿童医院 | 用于包装和表达变体因子viii以治疗血友病的改良表达组件 |
| EP3294309A4 (en) | 2015-05-14 | 2019-01-16 | St. Jude Children's Research Hospital, Inc. | NUCLEIC ACID MOLECULES WITH SPACERS AND METHOD FOR USE THEREOF |
| CA3001594A1 (en) * | 2015-10-14 | 2017-04-20 | Audentes Therapeutics, Inc. | Nucleic acid molecules containing spacers and methods of use thereof |
-
2016
- 2016-10-14 CA CA3001594A patent/CA3001594A1/en active Pending
- 2016-10-14 AU AU2016338565A patent/AU2016338565B2/en active Active
- 2016-10-14 WO PCT/US2016/057055 patent/WO2017066579A1/en not_active Ceased
- 2016-10-14 US US15/768,071 patent/US11230720B2/en active Active
- 2016-10-14 EP EP16856281.7A patent/EP3361869B8/en active Active
- 2016-10-14 PT PT168562817T patent/PT3361869T/pt unknown
- 2016-10-14 PL PL16856281.7T patent/PL3361869T3/pl unknown
-
2022
- 2022-01-21 US US17/581,203 patent/US12600986B2/en active Active
- 2022-02-02 AU AU2022200678A patent/AU2022200678B2/en active Active
-
2025
- 2025-01-31 AU AU2025200686A patent/AU2025200686A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US20180305715A1 (en) | 2018-10-25 |
| EP3361869B1 (en) | 2025-12-03 |
| EP3361869B8 (en) | 2026-01-14 |
| US20220411819A1 (en) | 2022-12-29 |
| US11230720B2 (en) | 2022-01-25 |
| AU2022200678A1 (en) | 2022-02-24 |
| WO2017066579A1 (en) | 2017-04-20 |
| AU2022200678B2 (en) | 2024-11-07 |
| EP3361869A1 (en) | 2018-08-22 |
| CA3001594A1 (en) | 2017-04-20 |
| AU2016338565A1 (en) | 2018-04-26 |
| US12600986B2 (en) | 2026-04-14 |
| PT3361869T (pt) | 2026-03-20 |
| AU2025200686A1 (en) | 2025-04-24 |
| EP3361869A4 (en) | 2019-04-03 |
| AU2016338565B2 (en) | 2021-11-18 |
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